Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis-Reference-Cited by-同舟云学术

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Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Published:2012-09 Issue:1-2 Volume:107 Page:213-221
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Hu Jie,Lu Jui-Yun,Wong Andrew M.S.,Hynan Linda S.,Birnbaum Shari G.,Yilmaz Denis S.,Streit Barbara M.,Lenartowicz Ewelina M.,Thompson Thomas C.M.,Cooper Jonathan D.,Hofmann Sandra L.

Funder

Taylor's Tale

Batten Disease Support and Research Association

National Institute for Neurological Disorders and Stroke

Batten Disease Family Association

Natalie Fund

Bletsoe Family

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference44 articles.

1. Mutations in the palmitoyl protein thioesterase gene causing infantile neuronal ceroid lipofuscinosis;Vesa;Nature,1995

2. Thematic review series: lipid posttranslational modifications. Lysosomal metabolism of lipid-modified proteins;Lu;J. Lipid Res.,2006

3. Update of the mutation spectrum and clinical correlations of over 360 mutations in eight genes that underlie the neuronal ceroid lipofuscinoses;Kousi;Hum. Mutat.,2012

4. Molecular genetics of palmitoyl–protein thioesterase deficiency in the U.S;Das;J. Clin. Invest.,1998

5. Adult neuronal ceroid lipofuscinosis with palmitoyl-protein thioesterase deficiency: first adult-onset patients of a childhood disease;van Diggelen;Ann. Neurol.,2001

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